• Announced the submission of a marketing application
  • to the European Medicines Agency (EMA) for a gene therapy.
  • Read more.

Latest news


The 30 members of the Telethon Scientific Committee on June 20-21 gave their final assessment of 160 projects that have passed...


This year, the call is dedicated to studies focused on a neglected disorder of genetic origin with a phenotype that is primarily expressed in bone, skin and subcutaneous tissues, or in the kidney. Read more.